Heart failure & cardiomyopathiesRandomised controlled trial
Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
- Randomised controlled trial
- double-blind
- placebo-controlled
- hierarchical testing
Authors' conclusion
Among patients with ATTR-CM, treatment with vutrisiran led to a lower risk of death from any cause and cardiovascular events than placebo and preserved functional capacity and quality of life.
Study context
- Population
- Patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)
- Participants
- 655 patients
- Comparison
- Vutrisiran vs placebo
- Vutrisirann = 326
- Placebon = 329reference
- Primary endpoint
- Composite of:
- All-cause death
- Recurrent CV events
Primary result
As reported in the abstract
Vutrisiran
125
events
Placebo
159
events
Vutrisiran vs PlaceboHazard ratio (95% CI)P value
overall population
0.72(0.56–0.93)
P = 0.01monotherapy population (no tafamidis at baseline)
0.67(0.49–0.93)
P = 0.02Secondary outcomes
| Outcome | HR (95% CI) | P value | |
|---|---|---|---|
| All-cause death · through 42 months · overall population | 0.65 (0.46–0.90) | P = 0.01 | |
| 0.512Hazard ratio (log scale) |
| Outcome | least-squares mean difference (m) (95% CI) | P value | |
|---|---|---|---|
| Change in 6-minute walk distance · overall population; less decline with vutrisiran | 26.5 (13.4–39.6) | P < 0.001 | |
| −2002040Difference, m (linear scale) |
| Outcome | least-squares mean difference (points) (95% CI) | P value | |
|---|---|---|---|
| Change in KCCQ-OS score · overall population; less decline with vutrisiran | 5.8 (2.4–9.2) | P < 0.001 | |
| −50510Difference, points (linear scale) |
Secondary end points, monotherapy population
- Similar benefits observed in the monotherapy population
Safety
Adverse events · similar in the two groups
Serious adverse events
Original publication
Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. Published 30 August 2024. doi:10.1056/nejmoa2409134
Summarised from the published abstract; every number is checked against it